Usage Examples
- The iPSP goes in within 60 days of the end-of-Phase 2 meeting, so the pediatric formulation decision cannot wait for the Phase 3 readout.
- FDA's written response asked for two more age cohorts, so the Agreed Initial Pediatric Study Plan slipped a cycle.
- The target is substantially relevant to a pediatric cancer, so orphan designation does not get us out of the investigation.
What is Pediatric Study Plan (PSP)?
Pediatric Study Plan is the FDA-agreed development document a sponsor submits before its NDA or BLA, outlining the pediatric studies it will run, their age groups and endpoints, and any deferral or waiver request.
The Pediatric Study Plan exists to force pediatric development into the program while the program can still change. Under 21 U.S.C. 355c(a)(1), an application for a new active ingredient, new indication, new dosage form, new dosing regimen, or new route of administration must carry pediatric assessments. The PSP is where the sponsor commits to producing them, before the marketing application is written.
The Pediatric Study Plan covers the planned pediatric studies and their objectives, design, age groups, endpoints, and statistical approach, plus any deferral, partial waiver, or full waiver the sponsor intends to request. The PSP does not replace the pediatric assessments themselves, and it does not lock the sponsor in: under 21 U.S.C. 355c(e)(4) either FDA or the applicant can amend the agreed plan at any time.
The Pediatric Study Plan runs on a fixed statutory clock in practice. The sponsor files the initial PSP no later than 60 calendar days after the end-of-Phase 2 meeting. FDA then has 90 calendar days to meet or respond in writing. The sponsor has a further 90 calendar days to file the Agreed Initial Pediatric Study Plan, which FDA confirms in writing within 30 calendar days.
Not to be confused with
- PIP
- the PIP is the European Union's pediatric planning instrument, agreed with EMA. The PSP is the separate US filing required under 21 U.S.C. 355c(e) and agreed with FDA, so a plan agreed in Europe does not discharge the US obligation.
- IND
- the IND authorizes the clinical program. The PSP commits to a pediatric development program inside it, and its deadline is keyed to the end-of-Phase 2 meeting, not to when the IND opened.
- Pediatric assessment
- the assessment is the study data itself, required with the application under 21 U.S.C. 355c(a)(2). The PSP is only the plan to generate that data; an agreed PSP satisfies the planning duty, not the assessment duty.
- Orphan drug designation
- orphan designation is a separate incentive status. It does not automatically remove pediatric obligations: 21 U.S.C. 355c(k) keeps the section applicable to an orphan-designated indication when the molecularly targeted pediatric cancer investigation applies.
The PSP obligations sit in the statute rather than in a single regulation, and these are the anchors.
What you must do
- 1Submit the initial pediatric study plan before the pediatric assessments or the molecularly targeted investigation, and no later than 60 calendar days after the end-of-Phase 2 meeting21 U.S.C. 355c(e)(2)(A)
- 2Include an outline of each planned pediatric study covering objectives and design, age groups, relevant endpoints, and statistical approach to the extent practicable21 U.S.C. 355c(e)(2)(B)
- 3Include any deferral, partial waiver, or waiver request in the plan itself, together with its supporting information, rather than raising it later21 U.S.C. 355c(e)(2)(B)
- 4Document agreement within 90 calendar days of the FDA meeting or written response, in a submission marked "Agreed Initial Pediatric Study Plan"; FDA confirms in writing within 30 calendar days21 U.S.C. 355c(e)(3)
- 5Certify the grounds for any full waiver, which FDA grants only where the applicant certifies and FDA finds the necessary studies are impossible or highly impracticable21 U.S.C. 355c(a)(5)
- 6For an adult oncology product directed at a molecular target substantially relevant to the growth or progression of a pediatric cancer, plan a molecularly targeted pediatric cancer investigation yielding dosing, safety, and preliminary efficacy data in appropriate formulations for each age group21 U.S.C. 355c(a)(3)
Common mistakes
Treating the 60-day filing date as the deadline that matters
the statute builds three further steps on top of it: FDA's meeting or written response, the sponsor's Agreed Initial Pediatric Study Plan, and FDA's written confirmation. The date the program actually depends on is the agreement date, and a thin initial outline pushes that date out by a full review cycle.
Assuming orphan designation ends the conversation
21 U.S.C. 355c(k) keeps the section applicable to an orphan-designated indication where the molecularly targeted pediatric cancer investigation under 355c(a)(3) applies. Oncology sponsors carrying a pre-RACE mental model find the obligation late, after the target is characterized and the ongoing adult program has no room left to absorb a pediatric arm.
Requesting a waiver without the certification
21 U.S.C. 355c(a)(5) grants a full waiver only where the applicant certifies and FDA finds the necessary studies impossible or highly impracticable. A paragraph asserting that the disease is rare in children, with no certification and no supporting information, is not a waiver request. It is an incomplete PSP section that comes back in FDA's response and restarts the agreement clock.
When This Matters
- The iPSP goes in within 60 days of the end-of-Phase 2 meeting, so the pediatric formulation decision cannot wait for the Phase 3 readout.
- FDA's written response asked for two more age cohorts, so the Agreed Initial Pediatric Study Plan slipped a cycle.
- The target is substantially relevant to a pediatric cancer, so orphan designation does not get us out of the investigation.
Frequently Asked Questions
The initial Pediatric Study Plan is due no later than 60 calendar days after the end-of-Phase 2 meeting, and always before the pediatric assessments or the molecularly targeted investigation are submitted, under 21 U.S.C. 355c(e)(2)(A). FDA then has 90 calendar days to meet with the sponsor or send a written response.
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